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NeuExcell’s NXL-004 Secures FDA Orphan Drug Designation for Glioma Treatment
On December 7, 2023, Beijing time, NeuExcell Therapeutics announced that its proprietary AAV gene therapy product, NXL-004, designed for treating malignant glioma, has been awarded Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). This...
PackGene Biotech Receives Exclusive C+ Round Investment from SDIC Fund, and Hosts a CGT Industry Salon to Discuss Global Strategies
December 1, 2023, PackGene Biotech, a frontrunner in gene therapy delivery, has successfully closed a landmark C+ funding round, securing over 100 million RMB exclusively from SDIC Fund Management Co., Ltd. This funding, a remarkable accomplishment in the current...
Tauopathy’s Role in Aβ Oligomer Formation: New Insights from Transgenic Primate Models on Alzheimer’s Pathogenesis
Tauopathy, characterized by protein tau and Aβ oligomer accumulation, is a significant aspect of Alzheimer's disease. The causal relationship between these accumulations and neurodegeneration remains unclear despite their links to various cognitive and movement...
Targeted Lysosomal Degradation of Mutant Huntingtin by Engineered Intrabody Peptide SM3
Huntington's disease (HD) is a devastating neurodegenerative condition caused by a mutation in the Huntingtin gene (HTT). This leads to the production of a mutant form of the huntingtin protein (mHTT) with an extended polyglutamine (polyQ) repeat, which has a...
Researchers Unveil a Novel Strategy for Protein Miniaturization in the CRISPR-Cas System
In a groundbreaking study published in Nature Communications, a team of researchers from China have introduced an innovative approach to protein miniaturization within the gene-editing realm of the CRISPR-Cas system. The CRISPR-Cas system is celebrated for its...
PackGene Biotech and Kudo Biotechnology Partner to Offer Customized mRNA Manufacturing Services
Collaboration provides end-to-end coverage of mRNA drug and vaccine development and manufacturing workflow HOUSTON, TX and NEEDHAM, MA – PackGene Biotech (PackGene) and Kudo Biotechnology (Kudo Bio) have partnered to provide high-quality, customized messenger RNA...
Zolgensma’s Success in SMA Treatment Advocates for AAV-based Gene Therapy and Promises a Bright Future for Genetic Disorders
At the 2023 Muscular Dystrophy Association Clinical and Scientific Conference, Novartis presented new data highlighting the continued efficacy and durability of its gene therapy treatment, Zolgensma, in treating spinal muscular atrophy (SMA). Zolgensma has been shown...
International Rare Disease Day | PackGene GMP plasmid is DMF registered, helping drug applications and shining lights on rare disease therapy!
February 28th is the 16th International Rare Disease Day with the theme of "Shine lights on your life" . Rare diseases, also known as "orphan diseases", have a low incidence rate and multiple disease types. Most of them are chronic, lifelong, severe, and...
Webinar: Treating Autosomal Recessive Deafness 9 with AAV1-hOTOF Gene Therapy (4/24/2024 10:00am EST/ 7:00am PST)
Abstract:Discover a groundbreaking advancement in treating Autosomal Recessive Deafness 9 (ARD9) with Dr. Yilai Shu, a pioneer in gene therapy for hearing loss. This webinar delves into the innovative AAV1-hOTOF gene therapy trial, offering new hope for individuals...
ASGCT (Maryland, May 7-11, 2024)
Abstract:Welcome to ASGCT 2024, the American Society of Gene and Cell Therapy's flagship conference. Esteemed researchers, clinicians, and industry leaders converge at this influential event to navigate the forefront of gene and cell therapy. In the vibrant atmosphere...
American Academy of Neurology (Colorado, Apri 13-18, 2024)
Abstract:The American Academy of Neurology Conference 2024 in Denver, a pivotal gathering for the brightest minds in neurology. This esteemed event brings together experts, researchers, clinicians, and innovators from around the globe to explore the forefront of...
Advanced Therapies Congress (London, Mar 19-20, 2024)
Abstract:The Advanced Therapies Congress convenes leaders in cell and gene therapy development across diverse sectors: pharma, biotech, startups, researchers, clinicians, academics, HTAs, payers, and regulators. In 2024, our new Gene Editing Track highlights CRISPR...
MDA (Florida, Mar 3-6, 2024)
Abstract:The Muscular Dystrophy Association (MDA) holds the top spot as the leading voluntary health organization in the United States, catering to individuals grappling with muscular dystrophy, ALS, and associated neuromuscular conditions. For over 70 years, MDA has...
Webinar: Overcoming Challenges in Preclinical NHP and Large Animal Studies for AAV-Based Gene Therapy through Improved AAV Purification and Quality Control Strategies
Abstract:Are you currently involved in the development of AAV-based gene therapy, specifically with non-human primates (NHP) or other large animals? Whether your focus is on constructing disease models or conducting pre-IND safety and toxicity studies, there are...
Webinar: Quality Delivery of mRNA – LNP with An Unbeatable Timeline
Abstract:Kudo Biotechnology is a global mRNA CDMO company headquartered in Singapore, providing world-class end-to-end cGMP manufacturing solutions for your mRNA needs. Kudo streamlines the manufacturing process of plasmid DNA, mRNA, lipid nanoparticle (LNP) and...
ESGCT 30th ANNUAL CONGRESS BRUSSELS
Time: 24-27 Oct 2023Address: Maison de la Poste, Bruxelles, Brussels, BelgiumContact: Irene Song (Sr. Director, Global Product) irene.song@packgene.comPoster P149:Generation of novel AAV serotype with enhanced infectivity, specificity, and lower toxicity via π-Icosa...
Voyager Therapeutics Advances Gene Therapy Candidate in Partnership with Neurocrine Biosciences
LEXINGTON, Mass., April 16, 2024-- Voyager Therapeutics, Inc. (Nasdaq: VYGR), today announced a significant milestone in its collaboration with Neurocrine Biosciences. The joint steering committee has selected a lead development candidate from the GBA1 gene therapy...
Biotech Innovators Nvelop Therapeutics and Seaport Therapeutics Launch with $100 Million to Revolutionize Genetic and Neuropsychiatric Treatments
In a significant development in the biotechnology sector, two pioneering companies, Nvelop Therapeutics and Seaport Therapeutics, have come out of stealth mode with a robust $100 million in financing each. This funding is set to propel their respective ventures into...
Exploring the role of RNA modification in normal hematopoiesis and acute myeloid leukemia pathogenesis
The review sheds light on the role of post-transcriptional RNA modifications in normal hematopoietic development and acute myeloid leukemia, and highlights novel therapeutic targets and treatment approaches. Credit: Chinese Medical JournalThe hematopoietic system is a...
Batavia, Chromatan Receive NIIMBL Grant to Improve AAV Manufacturing
Batavia Biosciences, a contract development and manufacturing organization, has partnered with ChromaTan, Inc. for the development and integration of BioRMB (formerly known as Continuous Countercurrent Tangential Chromatography or CCTC) technology into Batavia’s AAV...
New mRNA Cancer Vaccine Shows Promise in Pancreatic Cancer Treatment, MSK Study Reveals
In a groundbreaking development, researchers from Memorial Sloan Kettering Cancer Center (MSK) have presented new data that shines a hopeful light on the fight against pancreatic cancer. The innovative approach, employing the experimental messenger RNA (mRNA)-based...
AACR 2024: Geneos Therapeutics’ Vaccine Outperforms Liver Cancer Immunotherapies
SAN DIEGO, CA—“The future of cancer vaccines is now.” That’s the first thing that Geneos Therapeutics’ CEO and president, Niranjan Sardesai, PhD, said in an interview with GEN Edge at AACR 2024. “We’re at a point in time where there is no longer any doubt that...
Lisata Therapeutics Announces U.S. FDA Orphan Drug Designation Granted to LSTA1 for the Treatment of Osteosarcoma
Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, today announced that the U.S. Food and Drug...
Brain vesicles found to contain selectively packaged, full-length mRNA
This portrait of extracellular vesicles was taken using confocal laser scanning microscopy. The membrane was stained with fluorescent dye. Image titled "Exosomes" courtesy of Tomaž Einfalt, University of Basel, under Creative Commons BY-NC-ND license. Credit: Tomaž...
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